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    Summary
    EudraCT Number:2016-003038-26
    Sponsor's Protocol Code Number:ARGX-113-1603
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-12-27
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2016-003038-26
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia
    Estudio en fase II, aleatorizado, doble ciego y controlado con placebo para evaluar la seguridad, eficacia y farmacocinética de ARGX-113 en pacientes con trombocitopenia inmune primaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of the safety and effectiveness of ARGX-113 in Patients with Primary Immune Thrombocytopenia
    Un estudio sobre la seguridad y eficacia de ARGX-113 en pacientes con trombocitopenia inmune primaria
    A.4.1Sponsor's protocol code numberARGX-113-1603
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorArgenx BVBA
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportArgenx BVBA
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationArgenx BVBA
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7, Building C
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number+34961245875
    B.5.5Fax number+3293103499
    B.5.6E-mailregulatory@argenx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameARGX-113
    D.3.2Product code ARGX-113
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.3Other descriptive nameARGX-113
    D.3.9.4EV Substance CodeSUB180001
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Immune Thrombocytopenia
    Trombocitopenia inmune primaria
    E.1.1.1Medical condition in easily understood language
    Primary Immune Thrombocytopenia
    Trombocitopenia inmune primaria
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10074678
    E.1.2Term Primary immune thrombocytopenic purpura
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of ARGX-113.
    Evaluar la seguridad y tolerabilidad del ARGX-113.
    E.2.2Secondary objectives of the trial
    To evaluate the patients with initial response i.e., platelet count ≥ 30 × 109/L and/or at least doubling of the Baseline count and absence of bleeding.
    - To evaluate the clinical effect of ARGX-113 on:
    - platelet counts;
     - use of rescue treatment;
    - bleeding events.
    To assess the effect of ARGX-113 on quality of life.
    To assess the PK of ARGX-113.
    To assess the PD effects of ARGX-113.
    To evaluate the immunogenicity of ARGX-113.
    • Evaluar a los pacientes con respuesta inicial, es decir, recuento plaquetario ≥30 × 109/l o, como mínimo, duplicación del recuento plaquetario inicial y ausencia de hemorragia.
    • Evaluar el efecto clínico del ARGX-113 por medio de:
    o recuentos plaquetarios;
    o uso de tratamiento de rescate;
    o episodios hemorrágicos.
    • Evaluar el efecto del ARGX-113 en la calidad de vida.
    • Evaluar la farmacocinética (FC) del ARGX-113.
    • Evaluar los efectos farmacodinámicos (FD) del ARGX-113.
    • Evaluar la inmunogenicidad del ARGX-113
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ability to understand the requirements of the study, provide written informed consent (including consent for the use and disclosure of research-related health information), and comply with the study protocol procedures (including required study visits).
    2. Male or female patients aged ≥ 18 to ≤ 85 years.
    3. Eligible patients must receive standard-of-care treatment for ITP following the ASH guidelines and International Working Group (IWG) that has been stable in dose and frequency for at least 4 weeks prior to Screening. Standard-of-care may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist (see Section 7.9.2).
    4. Confirmed diagnosis of ITP according to the American Society of Hematology Criteria 2010 with blood platelet counts < 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.1 Note: The average platelet counts must be < 30 × 109/L with no single reading > 35 × 109/L as measured on 2 separate occasions at least 1 day apart during the Screening.
    5. Women of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline prior to administration of IMP.
    Women of childbearing potential are defined as all female patients unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with follicle-stimulating hormone (FSH) > 40 IU/L or are surgically sterile (i.e., women who had a hysterectomy, both ovaries surgically removed, or have current documented tubal ligation or any other permanent female sterilization procedure). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy if the value is within the post-menopausal range per the central laboratory.
    6. Female participants of childbearing potential must agree to use a highly effective method of birth control (i.e., pregnancy rate of less than 1% per year) during the study and for 90 days after the discontinuation of IMP.
    Contraceptive methods that result in a low failure rate when used consistently and correctly include methods such as combined hormonal contraception associated with inhibition of ovulation (oral, intravaginal, and transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, and implantable), intrauterine devices (IUDs), intrauterine hormone-releasing system, true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant-periodic abstinence [e.g., calendar, ovulation] and withdrawal methods are not acceptable methods of contraception), bilateral tubal occlusion, or a male partner who has had a vasectomy with documented aspermia post procedure. All female participants using hormonal contraceptive, especially those using mycophenolate, must also use a barrier method (i.e., condom or occlusive cap [diaphragm or cervical/vault caps]) and should have been stable on their hormonal contraceptive treatment for at least 4 weeks prior to Screening.
    7. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, of which 1 method must be a barrier method and the other another barrier method or highly effective form of contraception as described above for women of childbearing potential (e.g., condom with spermicidal cream or jelly, 1 hormonal plus 1 barrier method, IUD plus 1 barrier method or 2 simultaneous barrier methods). Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had vasectomy with documented aspermia post procedure can be included. In addition, male patients must be advised not to donate sperm during this period from signing of Informed Consent Form (ICF), throughout the duration of the study, and for 90 days after the last administration of IMP.
    1. Capacidad para comprender los requisitos del estudio, proporcionar el consentimiento informado por escrito (incluido el consentimiento por el uso y la divulgación de la información sanitaria relacionada con la investigación) y someterse a los procedimientos del protocolo del estudio (incluidas las visitas del estudio requeridas).
    2. Hombres o mujeres de ≥18 a ≤85 años de edad.
    3. Los pacientes aptos han de recibir un TE para la TIP, de conformidad con las directrices de la Asociación Estadounidense de Hematología (ASH) y el Grupo Internacional de Trabajo, con carácter estable en cuanto a dosis y frecuencia durante un mínimo de 4 semanas antes de la selección. El tratamiento estándar podrá incluir corticoesteroides orales, los inmunodepresores orales permitidos o un agonista del TPO-R (véase el apartado Sección 7.9.2).
    4. Diagnóstico confirmado de TIP de acuerdo con los criterios de 2010 de la ASH con recuentos plaquetarios en sangre <30 × 109/l y que no hayan sufrido una hemorragia grave en las últimas 4 semanas previas a la selección. Nota: Los recuentos plaquetarios promedio han de ser <30 × 109/l sin ninguna lectura >35 × 109/l, determinados en dos ocasiones distintas con al menos un día de diferencia durante la selección.
    5. Las mujeres en edad fértil deben presentar un resultado negativo en la prueba de embarazo en suero durante la selección y tener un resultado negativo en la prueba de embarazo en orina al inicio antes de la administración del PEI.
    Las mujeres en edad fértil se definen como todas las pacientes de sexo femenino a no ser que hayan llegado a la posmenopausia (definida como amenorrea continuada) durante al menos 1 año y con una hormona foliculoestimulante >40 IU/l o que sean quirúrgicamente estériles (es decir, que se hayan sometido a una histerectomía, una ooforectomía bilateral o que tengan documentada una ligadura de trompas o cualquier otro procedimiento de esterilización femenina permanente). La hormona foliculoestimulante puede usarse para confirmar el estado posmenopáusico en pacientes con amenorrea que no se encuentren en tratamiento de reemplazo hormonal si el valor está dentro del rango posmenopáusico según el laboratorio central.
    6. Las participantes en edad fértil deben estar de acuerdo en usar un método anticonceptivo altamente eficaz (es decir, con una tasa de embarazo de menos del 1 % al año) durante el estudio y 90 días tras la interrupción del PEI.
    Los métodos anticonceptivos que derivan en una tasa baja de ineficacia cuando se usan sistemática y correctamente incluyen métodos tales como anticoncepción hormonal combinada asociada a una inhibición de la ovulación (oral, intravaginal y transdérmica), anticoncepción hormonal basada solo en la progesterona asociada a una inhibición de la ovulación (oral, inyectable e implantable), dispositivos intrauterinos (DIU), sistema de liberación hormonal intrauterina, abstinencia sexual verdadera (cuando esto concuerda con el estilo de vida preferido y habitual de la participante; no se consideran métodos anticonceptivos aceptables la abstinencia periódica [p. ej., métodos de calendario, de ovulación] ni la marcha atrás), oclusión bilateral de trompas o un participante hombre que se haya sometido a una vasectomía con aspermia documentada posterior al procedimiento. Todas las participantes que usen un método anticonceptivo hormonal, en especial, las que usen micofenolato, deben usar también un método de barrera (es decir, preservativo o capuchón oclusivo [diafragma o capuchón cervical/en bóveda]) y haberse mantenido estables en el tratamiento anticonceptivo hormonal durante al menos las 4 semanas previas a la selección.
    7. Los pacientes de sexo masculino no esterilizados que sean sexualmente activos con una pareja de sexo femenino en edad fértil deben usar un método anticonceptivo doble efectivo, en el cual un método ha de ser uno de barrera y el otro, otro método de barrera o un método anticonceptivo altamente eficaz según lo descrito anteriormente para las mujeres en edad fértil (p. ej., preservativo con crema o gel espermicida, 1 método hormonal más 1 método de barrera, DIU más 1 método de barrera o 2 métodos de barrera simultáneamente). Pueden incluirse a hombres que practiquen una abstinencia sexual real (siempre que concuerde con el estilo de vida preferido y habitual del paciente). Podrá incluirse a pacientes de sexo masculino esterilizados que se hayan sometido a una vasectomía con aspermia documentada posterior al procedimiento. Además, se aconseja a los pacientes de sexo masculino que no donen esperma durante este periodo desde la firma del formulario de consentimiento informado, durante el transcurso del estudio y 90 días después de la administración del PEI.
    E.4Principal exclusion criteria
    1. Use of anticoagulants, or any drug with antiplatelet effect (e.g., acetylsalicylic acid [aspirin] or other salicylate containing medications, cyclooxygenase inhibitors, adenosine diphosphate (ADP) receptor inhibitors, phosphodiesterase inhibitors, glycoprotein IIB/IIIA inhibitors, adenosine reuptake inhibitors, thromboxane inhibitors, antimalarials, and prostacyclins) during the study and within 3 weeks prior to Screening.
    2. Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
    3. Use of IVIg or anti-D immunoglobulin treatment within 4 weeks prior to Screening.
    4. Use of recombinant thrombopoietin at any time.
    5. Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
    6. Use of corticosteroids which has not been stable for at least 4 weeks prior to Screening.
    7. Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine [up to 2.5 mg/kg/day], danazol [up to 15 mg/kg/day], mycophenolate mofetil [up to 3 g/day], mycophenolate sodium [up to 2160 mg/day]) which must have been stable for at least 4 weeks prior to Screening.
    8. Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
    9. Received vaccinations within 4 weeks prior to Screening or planned during the study.
    10. At Screening, have clinically significant laboratory abnormalities given as below:
    a. Aspartate aminotransferase (AST) or ALT > 3 × upper limit of normal (ULN).
    b. Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia as defined by the National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE], due solely to a documented medical diagnosis of Gilbert’s syndrome).
    c. Serum creatinine of > 1.5 mg/dL or creatinine clearance < 50 mL/min (using Chronic Kidney Disease Epidemiology - Creatinine formula).
    d. Hemoglobin ≤ 9 g/L.
    e. Thyroid stimulating hormone or thyroglobulin outside of the central laboratory normal range.
    f. International normalized ratio (INR) > 1.5 or activated partial thromboplastin time > 1.5 × ULN.
    g. Total immunoglobulin G (IgG) level < 6 g/L.
    h. Presence of ≥ 1+ proteinuria
    11. History of myeloproliferative or lymphoproliferative disorders at any time; or have a history of malignancy at any time unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years prior to Screening. Patients with completely excised nonmelanoma skin cancers or cervical carcinoma in situ would be permitted at any time.
    12. History of cerebrovascular accident or myocardial infarction within the last 12 months, before Screening, or current severe/unstable angina, arrhythmia, or at risk of ventricular arrhythmia, symptomatic congestive heart failure (New York Heart Association Class III or IV), or uncontrolled hypertension (Grade 3 to 4 CTCAE). Uncontrolled hypertension is defined as a repeated elevation in the blood pressure exceeding 140 mmHg and over 90 mmHg despite appropriate treatments.
    13. History of any thrombotic or embolic event within 12 months prior to Screening.
    14. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
    15. Known systemic lupus erythematosus, antiphospholipid antibody syndrome or any other auto-immune disease other than ITP.
    16. Prior history or symptoms suggestive of untreated Helicobacter pylori infection (recurrent nausea, vomiting, stomach burn, excessive burping, feeling bloated, and unexplained weight loss).
    17. History of a recent major surgery (that involve major organs e.g., brain, heart, lung liver, bladder, or gastrointestinal tract) within 4 weeks of Screening or that in the opinion of the investigator may compromise patient’s participation.
    **Please refer to the protocol on page 41 for the listed points 18 to 22.
    1. Uso de anticoagulantes o de cualquier fármaco con efecto antiplaquetario (p. ej., ácido acetilsalicílico [aspirina] u otros medicamentos que contengan salicilato, inhibidores de la ciclooxigenasa, inhibidores del receptor de la adenosina-difosfato, inhibidores de la fosfodiesterasa, inhibidores de la glicoproteína IIB/IIIA, inhibidores de la recaptación de la adenosina, inhibidores de los tromboxanos, antipalúdicos y prostaciclina) durante el estudio y en las 3 semanas previas a la selección.
    2. Pacientes que hayan recibido aportes de sangre o transfusiones en las 4 semanas previas a la selección.
    3. Uso de IgIV o tratamiento con inmunoglobulina anti D en las 4 semanas previas a la selección.
    4. Uso de trombopoyetina recombinante en cualquier momento.
    5. Uso de rituximab en los 6 meses previos a la selección. No se permite el uso de ningún anti CD20, salvo rituximab, en ningún momento.
    6. Uso de corticoesteroides que no se haya mantenido estable durante al menos las 4 semanas previas a la selección.
    7. No se permite el uso de inmunodepresores en las 4 semanas previas a la selección, a excepción de los siguientes inmunodepresores orales: azatioprina (hasta 2,5 mg/kg/día), danazol (hasta 15 mg/kg/día), micofenolato de mofetilo (hasta 3 g/día) y micofenolato sódico (hasta 2160 mg/día), que han de haberse mantenido estables durante al menos las 4 semanas previas a la selección.
    8. Uso de cualquier otro tratamiento biológico o fármaco en investigación distinto a los indicados anteriormente en los 3 meses o las 5 semividas del fármaco (lo que se prolongue más) previos a la selección.
    9. Recepción de vacunas en las 4 semanas previas a la selección o vacuna programada durante el estudio.
    10. En la selección, tener anomalías analíticas clínicamente significativas como se muestra a continuación:
    a. Aspartato aminotransferasa o alanina aminotransferasa >3 × límite superior de la normalidad (LSN).
    b. Bilirrubina sérica total >1,5 x LSN (excepto para la hiperbilirrubinemia de grado 1 según la definición de los Criterios terminológicos comunes para acontecimientos adversos [CTCAE] del Instituto Nacional del Cáncer de EE. UU., debida únicamente a un diagnóstico médico documentado de síndrome de Gilbert).
    c. Creatinina sérica >1,5 mg/dl o aclaramiento de creatinina <50 ml/min (usando la fórmula de la creatinina de epidemiología de la insuficiencia renal crónica).
    d. Hemoglobina ≤9 g/l.
    e. Hormona estimuladora de la tiroides o tiroglobulina fuera del rango normal del laboratorio central.
    f. Índice internacional normalizado >1,5 o tiempo de tromboplastina parcial activada >1,5 × LSN.
    g. Nivel total de inmunoglobulina G (IgG) <6 g/l.
    h. Presencia de proteinuria +≥1.
    11. Antecedentes de trastornos mieloproliferativos o linfoproliferativos en cualquier momento o antecedentes de neoplasia maligna en cualquier momento a no ser que se consideren curados con un tratamiento adecuado sin evidencia de recurrencia durante ≥5 años antes de la selección. Estarían permitidos en cualquier momento los pacientes con carcinomas cutáneos de tipo no melanómico o carcinoma cervical in situ.
    12. Antecedentes de accidente cerebrovascular o infarto de miocardio durante los últimos 12 meses antes de la selección o angina actual grave/inestable, arritmia, o riesgo de arritmia ventricular, insuficiencia cardíaca congestiva sintomática (de clase III o IV según la Asociación de Cardiología de Nueva York) o hipertensión no controlada (de grado 3 a 4 de los CTCAE). La hipertensión no controlada se define como una elevación repetida de la tensión arterial que supere los 140 mmHg y los 90 mmHg a pesar de los tratamientos adecuados.
    13. Antecedentes de episodios trombóticos o embólicos en los 12 meses previos a la selección.
    14. Antecedentes de coagulopatía o trombocitopenia hereditaria o antecedentes familiares de trombocitopenia.
    15. Conocimiento de lupus eritematoso sistémico, síndrome antifosfolipídico o cualquier otra enfermedad autoinmunitaria distinta a la TIP.
    16. Antecedentes o síntomas previos que sugieran una infección por Helicobacter pylori sin tratar (náuseas recurrentes, vómitos, ardor estomacal, exceso de eructos, sensación de hinchazón y pérdida de peso inexplicable).
    17. Antecedentes de cirugía mayor reciente (con implicación de los órganos principales, p. ej., cerebro, corazón, pulmones, hígado, vejiga o tracto gastrointestinal) en las 4 semanas previas a la selección o que, en opinión del investigador, pueda comprometer la participación del paciente.
    **Consulte el protocolo en la página 41 para los puntos enumerados 18 a 22.
    E.5 End points
    E.5.1Primary end point(s)
    -Incidence and severity of AEs (TEAEs) and serious AEs (SAEs).
    - Changes from Baseline (mean, median, minimum and maximum values, shifts) in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities, and clinical laboratory assessments.
    • Incidencia e intensidad de los acontecimientos adversos (AA) y acontecimientos adversos graves (AAG) surgidos durante del tratamiento.
    • Cambios con respecto al inicio (media, mediana, valores mínimos y máximos, cambios) en las constantes vitales, los parámetros electrocardiográficos (ECG), las anomalías en las exploraciones físicas y las evaluaciones analíticas clínicas.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Various time points throughout the study
    Varios puntos de tiempo durante todo el estudio
    E.5.2Secondary end point(s)
    -Frequency and proportion of patients with initial response i.e., platelet count ≥ 30 × 109/L and/or at least doubling of the Baseline count and absence of bleeding at any time during the study.
    - Mean change in platelet counts compared to Baseline.
    -Frequency and proportion of patients with following response at any time during the study 5,1:
    o Complete response (CR): Platelet count ≥ 100×109/L, confirmed on at least 2 separate occasions ≥ 7 days apart, and the absence of bleeding;
    o Response (R): Platelet count ≥ 30 and < 100×109/L, and a greater than 2-fold increase in platelet count from Baseline, confirmed on at least 2 separate occasions ≥ 7 days apart, and the absence of bleeding;
    o No response (NR): Platelet count < 30 × 109/L or less than doubling of the Baseline count or bleeding;
    -Time to initial response: time from starting treatment to time to reach CR or R;
    -Duration of response: time from the achievement of CR or R to loss of CR or R;
    -Response to ≥ 50×109/L: Platelet count increase to at least ≥ 50×109/L at any time during the study.
    - Frequency and proportion of patients requiring rescue therapy.
    -General bleeding assessment by the World Health Organization (WHO) bleeding scale and SMOG index of the ITP specific bleeding assessment tool (ITP-BAT).
    - Change from Baseline in the Short Form-36 item Health Status Questionnaire (SF-36) and the Functional Assessment of Cancer Therapy (FACT-Th6).
    - PK parameters of ARGX-113 including maximum observed concentration (Cmax), time of maximum concentration (tmax), concentration prior to dosing (Ctrough), apparent terminal half-life (t1/2,λz), and accumulation ratio (Rac).
    - Evaluation of pharmacodynamic markers: Total IgG, IgG isotypes IgG1, IgG2, IgG3, IgG4; and antiplatelet antibody levels. In addition, IgA, IgD, IgE, and IgM will also be assessed.
    - Evaluate the incidence of antidrug antibodies (ADA) to ARGX-113.
    • Frecuencia y proporción de pacientes con respuesta inicial, es decir, recuento plaquetario ≥30 × 109/l o, como mínimo, duplicación del recuento plaquetario inicial y ausencia de hemorragia en cualquier momento durante el estudio.
    • Cambio medio con respecto al inicio en los recuentos plaquetarios.
    • Frecuencia y proporción de los pacientes que presenten la siguiente respuesta en algún momento del estudio:
    - Respuesta completa (RC): recuento plaquetario ≥100 × 109/l, confirmado al menos en dos ocasiones distintas con ≥7 días de diferencia y ausencia de hemorragia.
    - Respuesta (R): recuento plaquetario ≥30 y <100 × 109/l y un aumento superior al doble con respecto al recuento plaquetario inicial, confirmado al menos en dos ocasiones distintas con ≥7 días de diferencia y ausencia de hemorragia.
    - Sin respuesta (SR): recuento plaquetario <30 × 109/l o inferior al doble del recuento plaquetario inicial o hemorragia.
    • Tiempo de respuesta: intervalo de tiempo desde el inicio del tratamiento hasta alcanzar una RC o R.
    • Duración de la respuesta: intervalo de tiempo desde que se alcanza una RC o R hasta que se pierde la RC o R.
    • Respuesta a ≥50 × 109/l: aumento del recuento plaquetario hasta, como mínimo, ≥50 × 109/l en cualquier momento del estudio.
    • Frecuencia y proporción de pacientes que requieran tratamiento de rescate.
    • Evaluación general de la hemorragia mediante la escala de hemorragia de la Organización Mundial de la Salud (OMS) e índice de piel, mucosa visible y órganos con gradación de gravedad (SMOG) de la herramienta de evaluación de la hemorragia específica de la TIP.
    • Cambio con respecto al inicio en el Cuestionario de salud resumido de 36 ítems y el Cuestionario de evaluación funcional para el tratamiento del cáncer.
    • Parámetros farmacocinéticos del ARGX-113, incluidos la concentración plasmática máxima observada (Cmáx), el tiempo de aparición de Cmáx (tmáx), la concentración plasmática observada previa a la dosis en las visitas 3, 5 y 7 (Cmín), la semivida terminal aparente, calculada a partir de (ln 2)/λz (solo en la visita 7) (t1/2, λz) y la tasa de acumulación, que se calcula como Cmáx del día 22/Cmáx del día 1 (Rac).
    • Marcadores FD: IgG total, isotipos de IgG (IgG1, IgG2, IgG3 e IgG4) y niveles de anticuerpos antiplaquetarios. Además, también se evaluarán la IgA, IgD, IgE, e IgM.
    • Incidencia de anticuerpos antifármaco (AAF) de ARGX-113.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Various time points throughout the study
    Varios puntos de tiempo durante todo el estudio
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 22
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 14
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 36
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None. Subject will continue with routine care.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-02-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-02-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-06-07
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